Positive data for AMO-02 in autism spectrum disorder

Privately-held AMO Pharma has announced an update on results of the recently-completed TIDE study of AMO-02 (tideglusib) in treatment of autism spectrum disorder (ASD) at the 65th Annual Meeting of the American Academy of Child and Adolescent Psychiatry in Seattle, Washington, USA.

This Phase II study was conducted at three Canadian clinical trial facilities and assessed the safety and efficacy of AMO-02, a novel orally available GSK3 beta inhibitor, in adolescents with ASD between the ages of 12 and 18 years-old (n = 83). The once-daily treatment for the core symptoms of ASD was found generally safe and well-tolerated, with adverse event rates that were generally similar between tideglusib and placebo. There were no treatment-associated serious adverse events.

Subjects in the Phase II randomized, 1:1 double-blinded study were treated with AMO-02 or placebo across a 12-week treatment period, with follow-up at four weeks. Daily dosing began at 400 mg and was increased incrementally up to 1,000mg based on the subject's weight. Patients treated with AMO-02 consistently outperformed placebo in measures of social withdrawal (ABC-Social) and repetitive behaviors (RBS-R), as well as daily living skills (Vineland), memory (NEPSY) and sleep quality (CSHQ). Outcome measures were based on measures including caregiver-and clinician-completed rating scales. A permutation test of efficacy results indicated that probability of false-positives was low.